There are many ocular diseases that affect vision. Diseases of the conjunctiva and cornea, cataracts, uveal diseases, retinal diseases, loss of central acuity and visual field abnormalities and diseases of Bruch's membrane are a few examples. Age-related macular degeneration is a “wet” form of age-related macular degeneration, choroidal neovascularization leads to progressive disease and vision loss.
Current therapeutics for treatment of many ocular conditions require the need for frequent intravitreal administration. Therapies involving delivery of proteins or aptamers are examples of such approaches, with the drawback that proteins and aptamers have short half-lives and require intravitreal administration every 4-6 weeks for life for maximal efficacy. Gene therapy approaches are potentially more long-term, with the possibility of lasting many months or years. Gene therapy can be in vivo, involving delivery of therapeutic genes directly to the tissue of interest, or can be ex vivo, where tissue selected for use is treated outside the body prior to implantation. The art has long sought gene therapy treatment methods that are safe for the patient and therapeutically viable.
One of the important factors in the efficacy and safety of gene therapy is the method used to introduce DNA into a cell. Viral vectors, such as retroviruses and adenoviruses, enable high expression of the introduced DNA but have safety concerns. Non-viral methods, such as liposomes, have low host immunogenicity but tend to suffer from inefficient DNA delivery to cells. Accordingly, there is a need in the art for new methods of introducing DNA into cells and tissues for the purpose of gene therapy.